Read through of truncated proteins in premature termination codon diseases by suppressor tRNAs

Patent metadata

Field	Value
Publication number	US-10982209-B2
Application number	US-201716083772-A
Country	US
Kind code	B2
Filing date	Mar 3, 2017
Priority date	Mar 10, 2016
Publication date	Apr 20, 2021
Grant date	Apr 20, 2021

Abstract

Provided are a method for constructing a suppressor tRNA, and 19 suppressor tRNAs corresponding to three termination codons, a plasmid, a vector or a kit comprising the above-mentioned tRNA. Also provided are use of the above-mentioned tRNA, plasmid, vector or kit in the manufacture of a medicament for treating a hereditary disease or a cancer caused by a nonsense mutation of a gene. Also provided are a method for evaluating the efficiency of a suppressor tRNA for reading through a nonsense mutation, and a method for restoring the expression of a truncated protein of a nonsense mutant of a pathogenic gene in a monogenic hereditary disease and a tumor suppressor gene in a tumor cell.

First claim

The invention claimed is:

1. A suppressor tRNA, wherein the suppressor tRNA is selected from the group consisting of the suppressor tRNAs set forth in SEQ ID NOs: 1-19.

2. The suppressor tRNA of claim 1 in a plasmid, a vector or a kit.

3. The kit of claim 2 , wherein the kit comprises a suppressor tRNA having the sequence set forth in any one of SEQ ID NOs: 1-19.

4. The kit of claim 3 , wherein the kit comprises the Amber suppressor tRNA (Gln) corresponding to SEQ ID NO: 1; the Ocher suppressor tRNA (Gln) corresponding to SEQ ID NO: 14; or the Opal suppressor tRNA (Arg) corresponding to SEQ ID NO: 7.

Assignees

• Univ Beijing

Inventors

• Xia Qing
• Wang Tianchang
• Yang Qi

Classifications

• A61P21/00 Primary

  Drugs for disorders of the muscular or neuromuscular system · CPC title

• C12N15/11

  DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology, C07H21/00); {Non-coding nucleic acids having a biological activity} · CPC title

• A61K48/00

  Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title

• C12N15/63

  Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression · CPC title

• A61P35/00

  Antineoplastic agents · CPC title